Status:
COMPLETED
A Study of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) in Japanese Participants With Primary Immunodeficiency Diseases (PID)
Lead Sponsor:
Baxalta now part of Shire
Conditions:
Primary Immunodeficiency Diseases (PID)
Eligibility:
All Genders
2+ years
Phase:
PHASE3
Brief Summary
In this study, Japanese participants with primary immunodeficiency diseases were treated with Immune Globulin Subcutaneous (Human), 20% solution, (IGSC, 20%). This study will be in 3 parts: Part 1: I...
Detailed Description
This study consists of 3 treatment parts (Epoch 1, 2, 3). The total evaluation period of the study will be 57 weeks in which screening period is for 2-8 weeks and Epoch 1 is from Week 8 to Week 21, Ep...
Eligibility Criteria
Inclusion
- Be of Japanese descent, defined as born in Japan and having Japanese parents and Japanese maternal and paternal grandparents.
- Participants must have a documented diagnosis of a form of primary humoral immunodeficiency involving antibody formation and requiring gammaglobulin replacement. The diagnosis must be confirmed by the medical director prior to treatment with IGIV.
- Participant is 2 years or older at the time of screening.
- Written informed consent is obtained from either the participants or the participants legally authorized representative prior to any study-related procedures and study product administration.
- Participant has been receiving a consistent dose of IGIV over a period of at least 3 months prior to screening equivalent to approximately 200-600 mg/kg-body weight (BW) per 3- 4 week period, as according to the product package insert
- Participant has a serum trough level of IgG \>= 5 gram per liter (g/L) at screening.
- Participant has not had a serious bacterial infection within the 3 months prior to screening.
- Participant is willing and able to comply with the requirements of the protocol.
Exclusion
- Participant has a known history of or is positive at screening for one or more of the following: hepatitis B surface antigen (HBsAg), polymerase chain reaction (PCR) for hepatitis C virus (HCV), PCR for human immunodeficiency virus (HIV) Type 1/2.
- Abnormal laboratory values at screening meeting any one of the following criteria (abnormal tests may be repeated once to determine if they are persistent):
- Persistent alanine aminotransferase (ALT) and aspartate amino transferase (AST) \> 2.5 times the upper limit of normal (ULN) for the testing laboratory
- Persistent severe neutropenia (defined as an absolute neutrophil count \[ANC\] \<= 500/milli cubic meter \[mm\^3\]).
- Participant has presence of renal function impairment defined by estimated glomerular filtration rate (eGFR) is \<60 milliliter per minute/ 1.73 square meter (mL/min/1.73m\^2).
- Participant has been diagnosed with or has a malignancy (other than adequately treated basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix), unless the disease-free period prior to screening exceeds 5 years.
- Participant is receiving anti-coagulation therapy or has a history of thrombotic episodes (including deep vein thrombosis, myocardial infarction, cerebrovascular accident, pulmonary embolism) within 12 months prior to screening or a history of thrombophilia.
- Participant has abnormal protein loss (protein losing enteropathy, nephrotic syndrome).
- Participant has anemia that would preclude phlebotomy for laboratory studies according to standard practice at the site.
- Participant has an ongoing history of hypersensitivity or persistent reactions (urticaria, breathing difficulty, severe hypotension, or anaphylaxis) following IV immunoglobulin, SC immunoglobulin, and/or Immune Serum Globulin (ISG) infusions.
- Participant has immunoglobulin A (IgA) deficiency (IgA less than 0.07 g/L), known anti IgA antibodies, and a history of hypersensitivity.
- Participant is on preventative (prophylactic) systemic antibacterial antibiotics at doses sufficient to treat or prevent bacterial infections, and cannot stop these antibiotics at the time of screening.
- Participant has active infection and is receiving antibiotic therapy for the treatment of infection at the time of screening.
- Participant has a bleeding disorder, or a platelet count less than 20,000/ microliter (mcL), or, in the opinion of the investigator, would be at significant risk of increased bleeding or bruising as a result of subcutaneous therapy.
- Participant has total protein \> 9 gram per deciliter (g/dL) or myeloma, or macroglobulinemia (IgM) or paraproteinemia.
- Women of childbearing potential meeting any one of the following criteria:
- Participant presents with a positive pregnancy test.
- Participant is breast feeding.
- Participant intends to begin nursing during the course of the study.
- Participant does not agree to employ adequate birth-control measures (e.g. intrauterine device, diaphragm or condom \[for male partner\] with spermicidal jelly or foam, or birth control pills/patches) throughout the course of the study.
- Participant has participated in another clinical study and has been exposed to an IP or device within 30 days prior to study enrollment.
- Participant is scheduled to participate in another non-observational (interventional) clinical study involving an IP or device during the course of the study.
- Participant has severe dermatitis that would preclude adequate sites for safe product administration.
Key Trial Info
Start Date :
August 11 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 22 2021
Estimated Enrollment :
17 Patients enrolled
Trial Details
Trial ID
NCT04346108
Start Date
August 11 2020
End Date
December 22 2021
Last Update
March 22 2024
Active Locations (8)
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1
Nagoya University Hospital
Nagoya, Aichi-ken, Japan, 466-8560
2
Kyushu University Hospital
Fukuoka, Fukuoka, Japan, 812-8582
3
Kurume University Hospital
Kurume-shi, Fukuoka, Japan, 830-0011
4
Gifu University Hospital
Gifu, Gifu, Japan, 501-1194